| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_f83734a45862157be1cc57b927717f5b |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2750-14143
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-14 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-713
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-86
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-713 |
| filingDate |
2020-09-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_eb7d5cc88ae45eb4eea820259dd68574
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_a3e17134ffb9cd7e84b5bcffb8e031dd
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_e541faddf35030f5c24b19e9103b590c
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4a2f5a2755915b43f44d04db134f8e42
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_5ca89c34aee596ed9bed4a19e0f882c8 |
| publicationDate |
2022-06-09-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
WO-2021053018-A9 |
| titleOfInvention |
Targeting misspliced transcripts in genetic disorders |
| abstract |
The present invention relates to repeat expansion disorders. Missplicing is understood to be a general phenomenon that can occur in repeat expansion disorders wherein the DNA and/or RNA sequence of repeat sequences in expanded repeat disorders can cause such aberrant transcription and/or aberrant splicing, resulting in misspliced transcripts, i.e. transcripts that do not have the putative splicing as observed e.g. for corresponding non-diseased genes. Such misspliced transcripts can be in particular associated with disease. Hence, the current invention now provides means and methods for targeting misspliced transcripts which is highly useful for the treatment of expanded repeat disorders. |
| priorityDate |
2019-09-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |